Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy

Abstract excerpt

Background Mucopolysaccharidosis type VI (MPS VI) is due to a deficiency of the lysosomal enzyme arylsulfatase B (ARSB) that results in multi-organ accumulation of glycosaminoglycans (GAGs). Limitations of current treatments prompted the development of a liver-directed gene therapy clinical trial for MPS VI. Methods We report the long-term follow-up of patients with MPS VI who discontinued enzyme replacement...

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